EU clears first mitochondrial disease therapy

Santhera Pharmaceuticals is celebrating the approval of its first product Raxone, which has been cleared in Europe for patients with a heritable genetic condition causing blindness, making it the region’s first therapy for a mitochondrial disease.

The European Commission green-lighted Raxone (idebenone) as the first treatment for visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON), an inherited mitochondrial disease which, if untreated, usually leads to rapid, profound and permanent blindness.

“Raxone represents a major breakthrough in mitochondrial disease treatment, and its approval paves the way for patients with LHON to be treated and to achieve a meaningful improvement of their visual acuity,” said Thomas Klopstock, MD, Professor for Neurology at the University of Munich.

The company said it is expecting to launch Raxone in Europe in the next few weeks, and that it plans to file the drug for approval in Duchenne Muscular Dystrophy on both sides of the pond later this year.